Patient safety must be paramount in new world of gene editing
- With the technique called CRISPR discovered by this year’s winners of the Nobel Prize for chemistry, it is now possible to envision treating many incurable diseases and correcting genetic defects before birth. But the public first needs to be reassured that transparent and stringent regulations and monitoring are in place
The Nobel Prize for chemistry rarely inspires so many explanatory pieces in the news media as the pioneering work of this year’s winners. It is not only that the prize is being shared by two women, rare in the annals of the Nobel, but the research of Emmanuelle Charpentier and Jennifer Doudna, who jointly published their seminal paper on gene editing less than a decade ago, has created explosive advances in biotechnology and biomedicine. Doudna was also the winner of last year’s Lui Che Woo Prize for Welfare Betterment in Hong Kong, where science honours such as the Shaw Prizes are dubbed Hong Kong’s Nobel prizes in terms of authority in recognising outstanding scientists around the world.
Since the discovery of DNA in the 1950s, scientists have been able to manipulate and tinker with the genomes of organisms. But their efforts were often crude and costly. With the technique called CRISPR discovered by the Nobel winners this year, gene editing has achieved precision and at a much lower cost than was possible previously.
With CRISPR and other even more advanced gene-editing techniques it has spawned, it is now possible to envision treating many incurable diseases, correcting genetic defects before birth and genetically disabling harmful pathogens.
But if people have fretted about the dangers of genetically modified food, they will be even more alarmed about genetically altered humans. In what was billed as the “first-ever CRISPR gene editing within [a] human body”, doctors at the Oregon Health and Science University in March used the technique to treat an eye patient in the hope of reversing his condition called Leber congenital amaurosis, the most common cause of inherited childhood blindness. The results are forthcoming.
But perhaps they should have called it the first authorised CRISPR gene editing in a human. In December, biophysicist He Jiankui was jailed for three years and fined 3 million yuan (US$447,000) in China for illegally using CRISPR in an operation to edit the genes of a pair of twins while still in embryo in an attempt to make them resistant to HIV. The twins’ father was HIV-positive.
The scandal alarmed the international scientific community. In response, Chinese regulators have enforced stringent guidelines and oversight. And in August, an international committee of experts declared that CRISPR was still not ready to experiment directly on human embryos. Among those who have been tireless in educating the public about the promise and peril of the new technique has been Professor Doudna herself. Nevertheless, the public needs to be reassured that transparent and stringent regulations and monitoring are in place and that safety and benefit for patients must outweigh all other considerations in this brave new world.
